CO2022005641A2 - Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígado - Google Patents
Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígadoInfo
- Publication number
- CO2022005641A2 CO2022005641A2 CONC2022/0005641A CO2022005641A CO2022005641A2 CO 2022005641 A2 CO2022005641 A2 CO 2022005641A2 CO 2022005641 A CO2022005641 A CO 2022005641A CO 2022005641 A2 CO2022005641 A2 CO 2022005641A2
- Authority
- CO
- Colombia
- Prior art keywords
- liver
- treatment
- gene therapy
- specific gene
- therapy vectors
- Prior art date
Links
- 206010019860 Hereditary angioedema Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title 1
- 210000004185 liver Anatomy 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 229940009550 c1 esterase inhibitor Drugs 0.000 abstract 1
- 108091006086 inhibitor proteins Proteins 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/005—Enzyme inhibitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962935359P | 2019-11-14 | 2019-11-14 | |
| US202063016365P | 2020-04-28 | 2020-04-28 | |
| PCT/US2020/060337 WO2021097157A1 (en) | 2019-11-14 | 2020-11-13 | Treatment of hereditary angioedema with liver-specific gene therapy vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2022005641A2 true CO2022005641A2 (es) | 2022-05-20 |
Family
ID=73834586
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2022/0005641A CO2022005641A2 (es) | 2019-11-14 | 2022-04-29 | Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígado |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20230340078A1 (https=) |
| EP (1) | EP4058475A1 (https=) |
| JP (1) | JP2023503850A (https=) |
| KR (1) | KR20220098210A (https=) |
| CN (1) | CN114829391A (https=) |
| AU (1) | AU2020384294A1 (https=) |
| BR (1) | BR112022009279A2 (https=) |
| CA (1) | CA3161154A1 (https=) |
| CL (1) | CL2022001260A1 (https=) |
| CO (1) | CO2022005641A2 (https=) |
| IL (1) | IL292717A (https=) |
| MX (1) | MX2022005869A (https=) |
| TW (1) | TW202128733A (https=) |
| WO (1) | WO2021097157A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| CA3207268A1 (en) * | 2021-01-27 | 2022-08-04 | Spark Therapeutics, Inc. | Compositions and methods for treating hereditary angioedema |
| KR20240054958A (ko) * | 2021-06-17 | 2024-04-26 | 메이라지티엑스 유케이 Ii 리미티드 | Aav 제조 방법 |
| US20240408240A1 (en) | 2021-10-01 | 2024-12-12 | Biomarin Pharmaceutical Inc. | Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations |
| CN117947040A (zh) | 2022-10-31 | 2024-04-30 | 苏州荷光科汇生物科技有限公司 | 用于目的基因的表达盒及其应用 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| JPWO2003033710A1 (ja) | 2001-10-16 | 2005-02-03 | 独立行政法人産業技術総合研究所 | 新規ｎ−アセチルグルコサミン転移酵素、それをコードする核酸及びそれに対する抗体並びにこれらの癌若しくは腫瘍診断用途 |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| AU2003212708A1 (en) | 2002-03-05 | 2003-09-16 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| AU2003221733A1 (en) * | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| US10214731B2 (en) * | 2015-05-28 | 2019-02-26 | Cornell University | Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2019209770B2 (en) * | 2018-01-16 | 2025-07-31 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
| CN113164560A (zh) * | 2018-10-26 | 2021-07-23 | Vrije布鲁塞尔大学 | 改善基因疗法的新工具及其用途 |
| WO2021081280A1 (en) * | 2019-10-23 | 2021-04-29 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
-
2020
- 2020-11-13 JP JP2022527987A patent/JP2023503850A/ja active Pending
- 2020-11-13 AU AU2020384294A patent/AU2020384294A1/en not_active Abandoned
- 2020-11-13 US US17/777,690 patent/US20230340078A1/en not_active Abandoned
- 2020-11-13 TW TW109139830A patent/TW202128733A/zh unknown
- 2020-11-13 CA CA3161154A patent/CA3161154A1/en active Pending
- 2020-11-13 CN CN202080087888.0A patent/CN114829391A/zh active Pending
- 2020-11-13 KR KR1020227019436A patent/KR20220098210A/ko active Pending
- 2020-11-13 WO PCT/US2020/060337 patent/WO2021097157A1/en not_active Ceased
- 2020-11-13 EP EP20824369.1A patent/EP4058475A1/en not_active Withdrawn
- 2020-11-13 BR BR112022009279A patent/BR112022009279A2/pt not_active IP Right Cessation
- 2020-11-13 MX MX2022005869A patent/MX2022005869A/es unknown
-
2022
- 2022-04-29 CO CONC2022/0005641A patent/CO2022005641A2/es unknown
- 2022-05-03 IL IL292717A patent/IL292717A/en unknown
- 2022-05-13 CL CL2022001260A patent/CL2022001260A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| BR112022009279A2 (pt) | 2022-09-06 |
| CN114829391A (zh) | 2022-07-29 |
| CL2022001260A1 (es) | 2023-03-10 |
| US20230340078A1 (en) | 2023-10-26 |
| JP2023503850A (ja) | 2023-02-01 |
| AU2020384294A1 (en) | 2022-06-02 |
| IL292717A (en) | 2022-07-01 |
| WO2021097157A1 (en) | 2021-05-20 |
| KR20220098210A (ko) | 2022-07-11 |
| EP4058475A1 (en) | 2022-09-21 |
| TW202128733A (zh) | 2021-08-01 |
| MX2022005869A (es) | 2022-06-14 |
| CA3161154A1 (en) | 2021-05-20 |
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